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Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study.

Author: , Al-DhekriHasan, Al-MousaHamoud, Al-SaudBandar, AlbertMichael H, AlonsoLaura, AquinoVictor M, ArnaoutRand, BalashovDmitry, BarlogisVincent, BertrandYves, BhatiaMonica, BlancheStephane, BoelensJaap Jan, BoothClaire, BordonVictoria, BrediusRobbert G M, CavazzanaMarina, CourteilleVirginie, CowanMorton J, CzogalaWojciech, DaviesE Graham, DoguFigen, DvorakChristopher C, FasthAnders, FerruaFrancesca, FischerAlain, FormankovaRenata, FouyssacFanny, GalimbertiStefania, GenneryAndrew Richard, GozdzikJolanta, HeilmannCarsten, HoenigManfred, IkinciogullariAydan, JandaAles, KałwakKrzysztof, KelečićJadranka, KeoghSteven John, KrivánGergely, LaberkoAlexandra, LangeAndrzej, LankesterArjan C, LindemansCaroline A, LjungmanPer, MahlaouiNizar, MeytsIsabelle, MoshousDespina, MustilloPeter, NevenBenedicte, PortaFulvio, SchuetzCatharina, SchulzAnsgar S, SedlacekPetr, ShawPeter John, ShcherbinaAnna, SlatterMary Anne, SmartJoanne, SmithAngela R, SonciniElena, SpeckmannCarsten, SuarezFelipe, SullivanKathleen E, SundinMikael, VeysPaul, Wolska-KusnierzBeata

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

This international collaborative study of 130 patients with CD40L deficiency who underwent hematopoietic stem cell transplantation (HSCT) found that HSCT was curative with improved outcomes when performed before organ damage development. Myeloablative conditioning was associated with better survival, event-free survival, and disease-free survival. Further research is needed to compare the risks of HSCT with those of lifelong supportive therapy.
Paper Details　
Original Abstract of the Article :
BACKGROUND: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation...See full text at original site
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引用元：
https://doi.org/10.1016/j.jaci.2018.12.1010

データ提供：米国国立医学図書館（NLM）

A Cure for CD40 Ligand Deficiency: Hematopoietic Stem Cell Transplantation

The world of immunology is a fascinating realm where our body's defense mechanisms are constantly at work, protecting us from a myriad of threats. CD40 ligand (CD40L) deficiency, a rare inherited immune disorder, can leave individuals vulnerable to recurrent infections. This study investigates the effectiveness of hematopoietic stem cell transplantation (HSCT) as a curative treatment for this debilitating condition. The researchers conducted a retrospective study, carefully analyzing data from a large cohort of patients who underwent HSCT for CD40L deficiency. Their findings suggest that HSCT can be a life-saving treatment for this disorder, offering the potential for long-term survival and a chance to live a healthier life. This is like finding a precious oasis in the desert of immune deficiency, providing a source of hope and healing for individuals with this challenging condition.

A Beacon of Hope: Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency

This study highlights the potential of HSCT in treating CD40L deficiency, a debilitating immune disorder. The researchers found that HSCT can significantly improve survival rates and provide a chance for long-term remission. This is like finding a hidden spring in the desert, offering a source of life and hope for individuals struggling with this challenging condition. The study emphasizes the importance of early intervention and appropriate donor selection, ensuring the best possible outcomes for patients undergoing HSCT.

A Journey Towards Immunity: Understanding the Role of HSCT

This study underscores the importance of ongoing research and innovation in the field of immunology. The findings highlight the potential of HSCT in treating CD40L deficiency, a rare but debilitating immune disorder. This knowledge empowers healthcare professionals to provide more effective and personalized treatment strategies for individuals with rare genetic conditions, potentially improving their quality of life and ensuring a brighter future.

Dr.Camel's Conclusion

This study is a testament to the power of medical innovation in addressing complex and rare genetic conditions. HSCT offers a beacon of hope for individuals with CD40L deficiency, providing a potential cure and a chance for a healthier life. It's like finding a precious oasis in the vast desert of immune deficiency, offering a source of relief and a path towards a healthier future.

Date :
  1. Date Completed 2020-06-08
  2. Date Revised 2020-06-08
Further Info :

Pubmed ID

30660643

DOI: Digital Object Identifier

10.1016/j.jaci.2018.12.1010

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Languages

English

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